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Using a mouse model of MPS IIIB, we administered a modified NAGLU by injection into the left ventricle of the brain, bypassing the blood\u2013brain barrier. The modification consisted of a fragment of IGFII, which allows receptor-mediated uptake and delivery to lysosomes. The modified enzyme was taken up avidly by cells in both brain and liver, where it reduced pathological accumulation of heparan sulfate and other metabolites to normal or near-normal levels. The results suggest the possibility of treatment for MPS IIIB.\n                  <\/jats:p>","DOI":"10.1073\/pnas.1416660111","type":"journal-article","created":{"date-parts":[[2014,9,30]],"date-time":"2014-09-30T11:28:38Z","timestamp":1412076518000},"page":"14870-14875","update-policy":"https:\/\/doi.org\/10.1073\/pnas.cm10313","source":"Crossref","is-referenced-by-count":105,"title":["Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB"],"prefix":"10.1073","volume":"111","author":[{"given":"Shih-hsin","family":"Kan","sequence":"first","affiliation":[{"name":"Department of Pediatrics, Los Angeles Biomedical Research Institute at Harbor\u2013UCLA Medical Center, Torrance, CA 90502;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mika","family":"Aoyagi-Scharber","sequence":"additional","affiliation":[{"name":"Research and Drug Discovery and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Steven Q.","family":"Le","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Los Angeles Biomedical Research Institute at Harbor\u2013UCLA Medical Center, Torrance, CA 90502;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jon","family":"Vincelette","sequence":"additional","affiliation":[{"name":"Research and Drug Discovery and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kazuhiro","family":"Ohmi","sequence":"additional","affiliation":[{"name":"Department of Biological Chemistry, David Geffen School of Medicine, University of California, Los Angeles, CA 90095"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sherry","family":"Bullens","sequence":"additional","affiliation":[{"name":"Research and Drug Discovery and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daniel J.","family":"Wendt","sequence":"additional","affiliation":[{"name":"Analytical Chemistry, BioMarin Pharmaceutical, Inc., Novato, CA 94949; and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Terri M.","family":"Christianson","sequence":"additional","affiliation":[{"name":"Research and Drug Discovery and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pascale M. 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